Off-target immune response from modified mRNA impacts future development
The Nobel Prize-winning modification that prevents the innate immune system from recognizing injected mRNA as foreign and blocking transcription of the protein it encodes has been found on some occasions to cause ribosomal frameshifting.

TRV-045 demonstrates efficacy for both reversal and prevention of CIPN in mice
Rhythm Pharmaceuticals advances next-generation MC4R agonist toward clinic for hypothalamic obesity
Bsense Bio nominates dual Kv7.2/3 and TRPV1 ion channel modulator as clinical candidate for chronic pain
Discovery of a new BLT2 receptor antagonist for the treatment of inflammatory airway diseases
Voyager selects lead development candidate for SOD-mutated ALS gene therapy program

Off-target immune response from modified mRNA impacts future development
The Nobel Prize-winning modification that prevents the innate immune system from recognizing injected mRNA as foreign and blocking transcription of the protein it encodes has been found on some occasions to cause ribosomal frameshifting.
TRV-045 demonstrates efficacy for both reversal and prevention of CIPN in mice
At the recent American College of Neuropsychopharmacology meeting, Trevena Inc. presented preclinical data for the novel selective sphingosine 1-phosphate S1P1 receptor modulator TRV-045, being developed for the treatment of chemotherapy-induced peripheral neuropathy (CIPN).
Rhythm Pharmaceuticals advances next-generation MC4R agonist toward clinic for hypothalamic obesity
Rhythm Pharmaceuticals Inc. has presented preclinical data on its new drug candidate, RM-718, a next-generation melanocortin MC4 receptor (MC4R) agonist for weekly administration with a potential development path for hypothalamic obesity.
Bsense Bio nominates dual Kv7.2/3 and TRPV1 ion channel modulator as clinical candidate for chronic pain
Bsense Bio Therapeutics Ltd. has nominated BSEN-760, a dual Kv7.2/3 and TRPV1 ion channel modulator, as a clinical candidate for the treatment of chronic pain.
Discovery of a new BLT2 receptor antagonist for the treatment of inflammatory airway diseases
Leukotriene B4 (LTB4) stimulates the release of cytokines and mediators that participate in tissue inflammation and acts as a chemoattractant for neutrophils, eosinophils and monocytes at the inflammation site.
Voyager selects lead development candidate for SOD-mutated ALS gene therapy program
Voyager Therapeutics Inc. has selected a lead development candidate for its superoxide dismutase 1 (SOD1)-mutated amyotrophic lateral sclerosis (ALS) gene therapy program. The company anticipates filing an IND application for the candidate with the FDA in mid-2025.
Holiday notice
In accordance with the publishing schedule, BioWorld Science was not published on Wednesday, Dec. 6, 2023, and will not be published on Friday, Dec. 8, 2023.
Other news to note for Dec. 7, 2023
Additional early-stage research and drug discovery news in brief.
Biotheryx discloses new CDK protein degraders
Biotheryx Inc. has developed new proteolysis targeting chimeras (PROTACs) comprising a E3 ubiquitin ligase binding moiety covalently bonded to a CDK protein-targeting moiety through a linker.
IGFBP7 SNP tied to the reversal phenotype of amyotrophic lateral sclerosis
Researchers have defined an amyotrophic lateral sclerosis (ALS) reversal phenotype as having an initial diagnosis of ALS but subsequently showing a progressive and sustained clinical improvement, based on an unusual case they found.
MC2 receptor ligands reported in Radionetics Oncology patent
Radionetics Oncology Inc. has patented new melanocortin MC2 receptor ligands reported to be useful for the diagnosis and treatment of cancer.
GDU-952 improves atopic dermatitis-like symptoms in mice
Researchers from Guangdong University of Technology and collaborators recently published details on the preclinical characterization of GDU-952.

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Conferences

Longboard’s 5-HT2C superagonist yields positive results in preclinical models of epilepsy
Neurology/psychiatric
Serotonin 5-HT2 receptor agonists have shown efficacy in treating seizure disorders, such as epilepsy. At the American Epilepsy Society meeting this week, Longboard Pharmaceuticals Inc. presented data on their 5-HT2C receptor superagonist LP-352 for the treatment of epilepsy.
Favorable preclinical profile of investigational gene therapy for TLE reported
Gene therapy
Temporal lobe epilepsy (TLE) is the most common form of focal epilepsy characterized by recurrent seizures originating in the hippocampus and with sprouting of mossy fiber axons that contribute to new recurrent synaptic excitability in the dentate gyrus (DG).
Iama Therapeutics’ IAMA-6 shows promise in preclinical mesial temporal lobe epilepsy
Neurology/psychiatric
Intracellular chloride levels are crucial for physiological brain development and correct functioning. On the other hand, aberrant expression of chloride importer NKCC1 (basolateral Na-K-Cl symporter, SLC12A2) and exporter KCC2 (K-Cl cotransporter 2, SLC12A5) is involved in neurodevelopmental and...
Bifunctional molecule capsid modulator and cGAS agonist achieves complete cure in AAV-HBV mice
American Association for the Study of Liver Diseases
LW-231, under development by Shanghai Longwood Biopharmaceuticals Co. Ltd., is a novel bifunctional molecule designed to simultaneously modulate hepatitis B virus (HBV) capsid assembly and activate cGAS-STING pathway. Preclinical data were recently presented.

Cancer

Simcere’s USP1 inhibitor receives FDA clearance for clinical trials in solid tumors
Regulatory
Simcere Pharmaceutical Group Ltd.’s subsidiary Simcere Zaiming Pharmaceutical Co. Ltd. has received FDA clearance of an IND application for SIM-0501, an oral small-molecule inhibitor of ubiquitin-specific peptidase 1 (USP1), for advanced solid tumors.

Infection

Ebviously presents in vitro data on EBV vaccine candidate
Immune
Ebviously GmbH, a spin-off from Helmholtz Munich (HMGU), has presented new in vitro data for its Epstein-Barr virus (EBV) vaccine candidate EBV-001. Based on noninfectious virus-like particles (VLPs) derived from EBV, EBV-001 is designed as a highly immunogenic, multiantigen vaccine to prevent...

Dual Mcl-1 and Bcl-2 inhibition as promising approach for treating tuberculosis
Tuberculosis still kills a lot of people worldwide (1.6 million deaths per year). Previous findings demonstrated that induced myeloid leukemia cell differentiation protein Mcl-1 inhibitors reduced the growth of Mycobacterium tuberculosis in human macrophages, but when inhibiting multiple proteins...
New dual inhibitors of host proteases TMPRSS2 and CTSL/CTSB as potential anti-SARS-CoV-2 agents
Coronavirus
Current antiviral agents for COVID-19 treatment target viral proteins, which are susceptible to mutation during SARS-CoV-2 evolution. A potential strategy to fight emerging drug resistances is the development of compounds targeting host proteins that are indispensable for the viral life cycle.

Neurology/psychiatric

Rev-erbA-α agonist ameliorates multiple sclerosis in mice
New compound
Nuclear receptor subfamily 1 group D member (NR1D1), also known as Rev-erbA-α, is highly expressed in the liver, adipose tissue, brain and skeletal muscle.
NLS Pharmaceutics enters option agreement with Aexon Labs for dual orexin receptor agonist platform
NLS Pharmaceutics Ltd. has entered into an exclusive worldwide option agreement with Aexon Labs Inc.
Oryzon Genomics awarded grant to support preclinical development of ORY-4001 in ALS
Grant
Oryzon Genomics SA has been awarded a $498,690 grant by the ALS Association to support the regulatory preclinical development of ORY-4001, a highly selective histone deacetylase 6 (HDAC6) inhibitor, for amyotrophic lateral sclerosis (ALS).
Stathmin-2 is new player in the ALS gene-verse
Musculoskeletal
A multi-institutional research team has suggested that aberrant TDP-43 processing of the pre-mRNA of a microtubule-associated protein, stathmin-2 (STMN2), may be the primary contributor to amyotrophic lateral sclerosis (ALS).
Psychogenics licenses rights to ENT1 inhibitor for neuropsychiatric disorders
License
PGI Drug Discovery LLC, the drug discovery arm of Psychogenics Inc., has entered into an exclusive license agreement with F. Hoffmann-La Roche Ltd. to obtain global development, manufacturing and commercialization rights to RO-7117997. Psychogenics intends to evaluate RO-7117997 as a potential...

Endocrine/metabolic

Preveceutical Medical to ramp up preclinical diabetes and obesity dual gene therapy program
Gene therapy
Preveceutical Medical Inc. is working to accelerate the completion of its preclinical diabetes and obesity dual gene therapy program. The company is pursuing a dual-gene therapy strategy using in vitro validated Smart-siRNA sequences, which when paired with its proprietary bioresponsive...

Biomarkers

miR-92a confers protection against inflammation-driven neurodegeneration
Neurology/psychiatric
MicroRNAs (miRNAs) play an important role in modulating neuronal stress responses, but their impact on neuroprotection has been poorly studied. Multiple sclerosis (MS) is the most common inflammatory disease affecting the central nervous system. Researchers have recently attempted to identify...

Somatic Ras-MAPK activating variants linked to mesial temporal lobe epilepsy
Conferences
Researchers from The Brigham and Women's Hospital presented data from a study that aimed to evaluate the role of somatic variants in drug-resistant mesial temporal lobe epilepsy (MTLE). High-coverage whole-exome sequencing was conducted using DNA samples derived from the hippocampus and paired...
Researchers find de novo CAMK2B variant in patient with tetralogy of Fallot
Conferences
CAMK2B encodes calcium/calmodulin-dependent protein kinase type II subunit β, a kinase expressed in the brain that has an important role in synaptic plasticity. Genetic variations are associated with neurodevelopmental disorders.

Gastrointestinal

Esperion Therapeutics discovers new ACLY inhibitors
Patents
Esperion Therapeutics Inc. has described ATP citrate lyase (ACLY) inhibitors reported to be useful for the treatment of cancer and more.
ZHX2 promotes liver recovery after acute liver injury
Dysfunction in the mitochondria contributes to the development of acute liver injury (ALI). There is emerging evidence indicating the mitochondria is key to maintain liver homeostasis and survival; thus, controlling its functioning is important...
Study links microbiome, vitamin B12, tissue repair in ulcerative colitis
Microbiome
A laboratory technique used to generate pluripotent stem cells from any tissue, cellular reprogramming, has led a group of researchers to the discovery of a process that could have an impact on natural tissue repair.
Novel hDHODH inhibitor for inflammatory bowel disease described
Human dihydroorotate dehydrogenase (hDHODH) is a mitochondrial enzyme participating in de novo pyrimidine biosynthesis essential for T- and B-lymphocyte proliferation.
Investigators find lactic acid bacteria strain that alleviates IBS in rats
Irritable bowel syndrome (IBS) is a functional gastrointestinal disorder where visceral hypersensitivity (VH), which causes discomfort and negatively impacts the quality of life, is influenced by stressful events. In recent years, the use of...
Lerna Biopharma presents first-in-class GalNAc-siRNA therapeutic for treatment of liver diseases
Conferences
Researchers from Lerna Biopharma Pte. Ltd. (formerly Cargene Therapeutics Pte. Ltd.) recently presented the discovery and preclinical evaluation of a first-in-class GalNAc-siRNA therapeutic, CG-LR1, being developed for the treatment of liver...

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